- Eidos grants Alexion exclusive license to
develop and commercialize AG10 in Japan -
- Phase 3 study of AG10 in ATTR cardiomyopathy
underway in U.S. & Europe; Phase 3 trial in ATTR polyneuropathy
planned to initiate in second half of 2019 -
- Agreement expands Alexion’s amyloidosis
portfolio -
- Eidos to receive upfront payment of $25
million and equity investment of $25 million, with potential for
additional Japanese-based milestone- & royalty-dependent
payments -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and BridgeBio
Pharma, Inc.’s (NASDAQ:BBIO) subsidiary Eidos Therapeutics, Inc.
(NASDAQ:EIDX) today announced an agreement that grants Alexion an
exclusive license to develop and commercialize AG10 in Japan. AG10
is a small molecule designed to treat the root cause of
transthyretin amyloidosis (ATTR) – destabilized and misfolded
transthyretin (TTR) protein – by binding and stabilizing TTR in the
blood. Eidos is currently evaluating AG10 in a Phase 3 study in the
U.S. and Europe for ATTR cardiomyopathy (ATTR-CM) – a progressive,
fatal disease caused by the accumulation of misfolded TTR amyloid
in the heart – and plans to begin a Phase 3 study in ATTR
polyneuropathy (ATTR-PN) – a progressive, fatal disease caused by
the accumulation of misfolded TTR amyloid in the peripheral nervous
system.
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“There is a significant need for new treatments for TTR
amyloidosis. We believe AG10 holds promise in its ability to
stabilize TTR and halt disease progression,” said John Orloff,
M.D., Executive Vice President and Head of Research &
Development at Alexion. “We are excited by the potential to grow
our amyloidosis portfolio by partnering with Eidos to expand the
development of AG10 to Japan. Alexion has more than 10 years of
experience operating there, and we look forward to applying our
expertise to bring AG10 to Japanese patients.”
“The Phase 2 study in ATTR-CM suggested that AG10 has the
potential to become an important treatment option for the
underserved ATTR-CM population. The trial showed that AG10 was
generally well-tolerated and resulted in near-complete
stabilization of TTR, which is known to be correlated with disease
severity in ATTR-CM. In the study, AG10 also normalized serum TTR
levels, a prognostic indicator of survival in ATTR patients,” said
Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of
Eidos. “We have now begun our Phase 3 program to evaluate the
safety and efficacy of AG10 in larger studies. This agreement
provides the potential opportunity to help even more patients
globally by leveraging Alexion’s significant development and
commercial experience to expand the AG10 program into Japan.”
Under the terms of the agreement, Alexion will acquire an
exclusive license for the clinical development and
commercialization of AG10 in Japan. Eidos will receive an upfront
payment of $25 million and an equity investment of $25 million at a
premium to the market price upon deal execution, with the potential
for additional Japanese-based milestone- and royalty-dependent
payments.
About AG10
AG10 is an investigational, orally-administered small molecule
designed to potently stabilize tetrameric transthyretin, or TTR,
thereby halting at its outset the series of molecular events that
give rise to TTR amyloidosis, or ATTR. In a Phase 2 clinical trial
in patients with symptomatic ATTR-CM, AG10 was generally well
tolerated, demonstrated greater than 90 percent average TTR
stabilization at Day 28, and increased serum TTR concentrations, a
prognostic indicator of survival in a retrospective study of
ATTR-CM patients, in a dose-dependent manner.
AG10 was designed to mimic a naturally-occurring variant of the
TTR gene (T119M) that is considered a rescue mutation because
co-inheritance has been shown to prevent or ameliorate ATTR in
individuals also inheriting a pathogenic, or disease-causing,
mutation in the TTR gene. To our knowledge, AG10 is the only TTR
stabilizer in development that has been observed to mimic the
stabilizing structure of this rescue mutation.
The Phase 3 ATTRibute-CM study of AG10 in patients with ATTR-CM
is underway in the United States and Europe. Part A of the study
will assess the change from baseline in 6-minute walk distance
(6MWD) at 12 months. Part B of the study will evaluate reduction in
all-cause mortality and frequency of cardiovascular-related
hospitalizations will be evaluated at 30 months. In addition, Eidos
plans to initiate a Phase 3 study of AG10 in ATTR polyneuropathy
(ATTR-PN) in the second half of 2019.
About Transthyretin Amyloidosis (ATTR)
There is significant medical need in transthyretin amyloidosis
(ATTR) given the large patient population and an inadequate current
standard of care. ATTR is caused by the destabilization of TTR due
to inherited mutations or aging and is commonly divided into three
distinct categories: wild-type ATTR cardiomyopathy (ATTRwt-CM),
mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy
(ATTR-PN). The worldwide prevalence of each disease is
approximately 400,000 patients, 40,000 patients and 10,000
patients, respectively.
All three forms of ATTR are progressive and fatal. For patients
with untreated ATTRwt-CM and ATTRm-CM, symptoms usually manifest
later in life (age 50+), with median survival of three to five
years from diagnosis. ATTR-PN either presents in a patient's early
30s or later (age 50+), and results in a median life expectancy of
five to ten years from diagnosis for untreated patients.
Progression of all forms of ATTR causes significant morbidity,
impacts productivity and quality of life, and creates a significant
economic burden due to the costs associated with progressively
greater patient needs for supportive care.
About Alexion
Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases through the
discovery, development and commercialization of life-changing
therapies. As the global leader in complement biology and
inhibition for more than 20 years, Alexion has developed and
commercializes two approved complement inhibitors to treat patients
with paroxysmal nocturnal hemoglobinuria (PNH) as well as the first
and only approved complement inhibitor to treat atypical hemolytic
uremic syndrome (aHUS), anti-acetylcholine receptor (AchR)
antibody-positive generalized myasthenia gravis (gMG) and
neuromyelitis optica spectrum disorder (NMOSD). Alexion also has
two highly innovative enzyme replacement therapies for patients
with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency
(LAL-D). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. Alexion focuses its research efforts on novel
molecules and targets in the complement cascade and its development
efforts on the core therapeutic areas of hematology, nephrology,
neurology, and metabolic disorders. Alexion has been named to the
Forbes’ list of the World’s Most Innovative Companies seven years
in a row and is headquartered in Boston, Massachusetts’ Innovation
District. The company also has offices around the globe and serves
patients in more than 50 countries. This press release and further
information about Alexion can be found at: www.alexion.com.
[ALXN-G]
About BridgeBio and Eidos
BridgeBio is a team of experienced drug discoverers, developers
and innovators working to create life-altering medicines that
target well-characterized genetic diseases at their source.
BridgeBio was founded in 2015 to identify and advance
transformative medicines to treat patients who suffer from
Mendelian diseases, which are diseases that arise from defects in a
single gene, and cancers with clear genetic drivers. BridgeBio’s
pipeline of over 15 development programs includes product
candidates ranging from early discovery to late-stage development.
For more information, please visit www.bridgebio.com.
Eidos is a BridgeBio Pharma subsidiary focused on addressing the
large and growing unmet need in diseases caused by transthyretin
(TTR) amyloidosis (ATTR). Eidos is developing AG10, a potentially
disease-modifying therapy for the treatment of ATTR. For more
information, please visit www.eidostx.com.
Forward-Looking Statements
This press release includes forward-looking statements. Such
forward-looking statements are subject to risks and uncertainties
that could cause actual results to differ materially from those
expressed or implied in such statements. Examples of
forward-looking statements include, among others, statements we
make regarding: (i) the therapeutic benefits and commercial
potential of AG10 in Japan and elsewhere; (ii) development plans
and clinical trial plans related to AG10; (iii) the potential of
AG10 for the treatment of ATTR cardiomyopathy, ATTR polyneuropathy
and other conditions; (iv) that the rights of Alexion under the
license agreement will result in growth of Alexion’s amyloidosis
portfolio by partnering with Eidos to expand the development of
AG10 to Japan; and (v) the likelihood that AG10 will be approved
for commercial sale in Japan. The process by which products such as
AG10 could potentially be developed and approved for commercial
sale is long and subject to highly significant risks. Applicable
risks and uncertainties include: results in early stage clinical
trials may not be indicative of full results or results from later
stage or larger clinical trials (or in broader patient populations)
and do not ensure regulatory approval; the possibility that results
of clinical trials are not predictive of safety and efficacy and
potency of products (or the failure to adequately operate or manage
our clinical trials) which could cause the halt of trials, delays
or prevention from making regulatory approval filings or result in
denial of regulatory approval of product candidates; unexpected
delays in clinical trials; unexpected concerns that may arise from
additional data or analysis obtained during clinical trials; delays
or failure of product candidates to obtain regulatory approval due
to clinical trial results, issues with clinical trial products,
unexpected expense or otherwise; as well as those additional risks
relating to product development and approval and other risks
identified under the heading "Risk Factors" included in Alexion’s,
BridgeBio’s and Eidos’ most recent Form 10-Q filings and in their
respective other future filings with the SEC. The forward-looking
statements contained in this press release reflect Alexion’s,
BridgeBio’s and Eidos’ current views with respect to future events.
Alexion, BridgeBio and Eidos do not undertake and each specifically
disclaims any obligation to update any forward-looking statements,
except as required by law.
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Alexion Contacts: Media Megan Goulart,
857-338-8634 Senior Director, Corporate Communications
Investors Susan Altschuller, Ph.D., 857-338-8788 Vice
President, Investor Relations Eidos Contacts: Media
Carolyn Hawley, Canale Communications 619-849-5382,
carolyn@canalecomm.com Investors John Grimaldi, Burns
McClellan 212-213-0006, jgrimaldi@burnsmc.com BridgeBio
Contact: Media Alberto Gestri
AGestri@theoutcastagency.com
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